Allos Pharma
Biopharmaceutical company advancing an investigational GABA-B receptor agonist as a potential disease-modifying treatment for autism spectrum disorder and Fragile X syndrome. The organization combines translational neuroscience, preclinical validation across multiple animal models, biomarker-driven human pharmacodynamic studies, and late-stage clinical development and regulatory engagement.
Industries
Nr. of Employees
small (1-50)
Products
Investigational GABA-B receptor agonist candidate (clinical-stage)
An investigational selective GABA-B receptor agonist in development as a potential disease-modifying treatment for autism spectrum disorder and Fragile X syndrome; has prior human safety data and been evaluated in Phase 2 and Phase 3 trials and open-label extensions.
Arbaclofen
A GABA-B receptor agonist evaluated in clinical trials for the treatment of Fragile X syndrome and autism, demonstrating meaningful improvements in core behavioral problem scores.
Investigational GABA-B receptor agonist candidate (clinical-stage)
An investigational selective GABA-B receptor agonist in development as a potential disease-modifying treatment for autism spectrum disorder and Fragile X syndrome; has prior human safety data and been evaluated in Phase 2 and Phase 3 trials and open-label extensions.
Arbaclofen
A GABA-B receptor agonist evaluated in clinical trials for the treatment of Fragile X syndrome and autism, demonstrating meaningful improvements in core behavioral problem scores.
Services
Partnerships for co-development, licensing, and investigator collaborations to advance an investigational CNS compound through confirmatory trials and regulatory interactions.
Partnerships for co-development, licensing, and investigator collaborations to advance an investigational CNS compound through confirmatory trials and regulatory interactions.
Expertise Areas
- Clinical trial management
- Translational neuroscience
- Pharmacodynamic biomarker development
- Regulatory strategy and engagement
Key Technologies
- GABA-B receptor modulation
- iPSC-derived neuronal electrophysiology
- Electroencephalography (EEG) including phase-amplitude coupling analysis
- Steady-state visual evoked potential (SSVEP) assays
News & Updates
White paper summarizing mechanistic, nonclinical, and human pharmacodynamic evidence supporting GABA-B receptor modulation for ASD; prepared for discussion with the U.S. FDA (September 2026).
Collection of peer-reviewed articles documenting preclinical validation, clinical trials, and human biomarker studies relevant to the development program.
Completed multiple formal FDA meetings and established alignment on a single confirmatory Phase 3 trial design for a genetically defined neurodevelopmental indication.
Acquisition of global rights to the investigational GABA-B receptor agonist to pursue development across neurodevelopmental indications.
Pre-NDA meeting with the U.S. FDA scheduled in support of an autism spectrum disorder development program.
White paper summarizing mechanistic, nonclinical, and human pharmacodynamic evidence supporting GABA-B receptor modulation for ASD; prepared for discussion with the U.S. FDA (September 2026).
Collection of peer-reviewed articles documenting preclinical validation, clinical trials, and human biomarker studies relevant to the development program.
Completed multiple formal FDA meetings and established alignment on a single confirmatory Phase 3 trial design for a genetically defined neurodevelopmental indication.
Acquisition of global rights to the investigational GABA-B receptor agonist to pursue development across neurodevelopmental indications.
Pre-NDA meeting with the U.S. FDA scheduled in support of an autism spectrum disorder development program.